Neurology

The founding president and chief executive officer of Parent Project Muscular Dystrophy discussed recent and upcoming milestones in the field.

Juan Francisco Cabello, MD, the head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile, discussed the current state of newborn screening and what needs to change.

The PDUFA data has been set for March 18, 2024.

The chief scientific officer of CureDuchenne discussed progress in the field so far and upcoming milestones.

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, spoke about how his lab’s findings may imply a need to rethink epilepsy treatment development.

The postdoctoral fellow and member of the La Spada Lab at University of California, Irvine, discussed findings he presented at ANA’s 2023 conference that may have implications for future RNA therapy development.

Review top news and interview highlights from the week ending September 15, 2023.

The head of the Pediatric Neurology Fellowship Program at the University of Valparaiso in Chile discussed how a lack of cost-effectiveness may hold back the potential of newborn screening in gene therapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Sattar Khoshkhoo, MD, the founding director of the Epilepsy Genetics Clinic at Brigham and Women's Hospital, discussed his nonclinical research on disease pathogenesis in MTLE.

The chief research officer, Muscular Dystrophy Association, shared a message for Duchenne Action Month.

The head of the Pediatric Neurology Fellowship Program at the University of Valparaíso in Chile discussed positive impact of newborn screening so far and work that still needs to be done.

Study author Mya C. Schiess, MD, commented on trends seen in the blinded findings so far.

ATA-100 is currently being evaluated in a multicenter phase 1/2 clinical trial (NCT05224505) in Denmark, France, and United Kingdom.

Review top news and interview highlights from the week ending September 8, 2023.

Significant improvements in ON time without troublesome dyskinesia were reported with the cell therapy after 1 year, with more apparent effects observed in the high-dose group.

In light of the IND clearance, uniQure announced its intention to carry out a phase 1/2a clinical trial, with screening of potential participants with refractory MTLE anticipated to begin in the last 3 months of 2023.

New data shows the ASO therapy’s benefit in older populations, following beneficial findings in patients previously treated with gene therapy.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

The data come from 30 patients treated across 2 clinical trials and 9 patients treated in expanded access frameworks who had follow-up times ranging from 0.64 years to 12.19 years.

Review top news and interview highlights from the week ending September 1, 2023.

Data from a multiple-ascending dose study suggest the investigational agent developed by Design Therapeutics was generally well tolerated with dose-related increases in frataxin levels.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

TSHA-102 has previously received orphan drug and rare pediatric disease designations from the agency and is being investigated in the REVEAL phase 1/2 trial (NCT05606614).

In light of the promising results, BlueRock is preparing for a phase 2 trial.

Pending approval, ARO-DUX4 will be evaluated in patients with facioscapulohumeral muscular dystrophy and ARO-SOD1 will be evaluated in patients with amyotrophic lateral sclerosis.

Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

Earlier this year Bloomsbury Genetic Therapies met with the UK’s MHRA for a scientific advice meeting to discuss BGT-NPC.

The codirector of the MDA Clinic and professor of neurology at Johns Hopkins discussed the importance of connecting infants with spinal muscular atrophy to a therapy as soon as possible.

BGT-OTCD is being developed in a collaboration between Bloomsbury Genetic Therapies and University College London.